Chinese patients living with pediatric growth hormone deficiency are one step closer to a new treatment option, according to a recent article from PharmaBiz.com. The step I’m referring to is positive data from a phase 3 trial of lonapegsomatropin (TransCon hGH) that was released in May.

This trial was active-controlled, randomized, and open-label with a primary endpoint of annualized height velocity (AHV) after 52 weeks of treatment. Children with growth hormone (GH) deficiency were dosed with lonapegsomatropin once a week for a year. At the end of the trial, researchers observed the following results:

• The study drug was superior to daily growth hormone treatment in relation to AHV
• Lonapegsomatropin was well-tolerated by patients
  • Its safety profile was comparable to that of daily growth hormone treatment
    • It was also consistent with previous research on lonapegsomatropin

This data is planned to be part of Visen Pharmaceuticals’ – the drug’s developer – Biologic License Application (BLA), which will be submitted to the National Medical Products Administration (NMPA) when all preparatory work is complete.

About GH Deficiency 

As the name suggests, growth hormone deficiency (GHD) occurs when the body makes insufficient amounts of growth hormone. When there isn’t enough of this hormone, the body is unable to grow at a normal rate, resulting in short stature. Other symptoms include:

• Fine hair
• Poor nail growth
• Stunted growth
• Hypoglycemia (this symptom is present in newborns)
• High pitched voice
• Small genitals (this symptom is present in males)
• Immature appearance
• “Chubby” build
• Slow tooth eruption
• Prominent forehead

These symptoms are all the result of too little growth hormone, which can be caused by many different things. These include genetic mutations (the POU1F1/Pit1, PROP1, GHRH, and GH1 genes have all been connected to GHD), brain trauma, radiation therapy, infection, and brain tumors. GHD is treated by injecting more of the growth hormone into affected individuals.